Duchenne muscular dystrophy (DMD) is a rare muscle disorder but it is one of the most frequent genetic conditions affecting approximately 1 in 3,500 male births worldwide. DMD is characterized by weakness and wasting (atrophy) of the muscles of the pelvic area followed by the involvement of the shoulder muscles.
There are about 12,000 patients with the disease in this country. Because it is a relatively rare disease, treatment is very expensive. How expensive? A drug that's decades old has just been approved for use for treatment of Duchene in the United States, but at the cost of a 7300% increase of the price outside of this country.
That means a years' treatment could cost $89,000 according to Consumer Reports (http://www.consumerreports.org/drug-prices/emflaza-drug-price-hike-questioned/) because it's covered under a well-intentioned law designed to promote drug development for orphan diseases.
That is, if its ever actually allowed on the market - as soon as it was made known the manufacturer intended to increase the cost that much, congress investigated...which led to a delay in getting the drug into the hands of those who really need it.
Imagine now, for a moment, that your loved one had a fatal disease and that there was a drug that could help them walk just a little longer....if they could just get access to it.
Meanwhile, right here in Massachusetts a drug company has developed a - drug specifically for treatment of DMD - http://jettfoundation.org/news/fda-grants-accelerated-approval-to-first-drug-for-duchenne-muscular-dystrophy/ - so awareness is paying off, and drug development is happening.
I'm raising money to raise awareness of this disease and to help promote research so that Duchene patients and their families aren't held hostage to Marathon Pharmaceuticals and in hope that other, alternatives can be developed.
You, my friends, know I periodically raise money for causes I care about: LUK, Inc supporting foster care programs, a backpacks for veterans program my kids put together, The O'Connell Valor Fund giving 99.9% of all donations directly to Veterans care. I don't take asking you to support these causes lightly, and this is no different.
Please consider a modest donation to my effort. The payoff is participation in the Cohasset Triathalon representing the Jett Foundation.
Since 2001, the Jett Foundation, located in Kingston, MA, has worked to find treatments and a cure for Duchenne muscular dystrophy (DMD) while improving the lives of those affected by DMD. Over the past decade, the Jett Foundation has raised almost $6 million dollars for Duchenne research.
The Jett Foundation also provides resources for treatments and adaptive activities as we work toward a cure and treatments for Duchenne; the foundation funds the Jett Program for Pediatric Neuromuscular Disorders (JPPND) at Mass General Hospital for Children (MGHfC), and Camp Promise. Additionally, the foundation provides advocacy services for families affected by Duchenne.
The Jett Foundation is a registered charity with 501(c)(3) status from the IRS; all donations are tax deductible.